Woman Back on Dialysis After Doctors Remove Failing Pig Kidney
Xenotransplantation, using gene-edited pig kidneys, faced challenges in managing blood flow and immune response, leading to early organ removal. [ more ]
Controversial scientist says he'll use gene editing to end HIV & Alzheimer's
Controversial Chinese scientist He Jiankui faces consequences for gene-editing experiments on twins.
Despite criticism, He now aims to use 'base editing' to prevent Alzheimer's disease, setting the stage for potential breakthroughs in curing other diseases. [ more ]
A Gene-Edited Pig Liver Was Attached to a Person-and Worked for 3 Days
Scientists used gene editing to make 69 genetic edits to a pig, including knocking out three pig genes and inserting seven human genes.
Previous attempts with unmodified pig livers failed within hours, but the pig livers with genetic edits in the Penn study showed stable blood flow and no signs of inflammation. [ more ]
Gene-editing technology CRISPR has successfully treated a patient with sickle-cell disease, providing hope for future treatments.
The treatment resulted in the patient living virtually symptom-free, with 29 out of 30 eligible patients experiencing zero pain crises after treatment. [ more ]
UK authorizes first gene therapy for treating sickle cell disease
The UK's MHRA has approved the use of a gene-editing therapy for patients with sickle cell disease and beta thalassemia.
The therapy, called Casgevy, is the first-ever approved therapy that uses CRISPR-based gene editing technology.
The therapy has shown high efficacy in relieving severe pain associated with these blood disorders and could potentially replace the current standard of care. [ more ]
A Single Infusion of a Gene-Editing Treatment Lowered High Cholesterol
Researchers have shown that a single infusion of a gene-editing treatment can reduce cholesterol levels in people.
The treatment aims to permanently lower cholesterol by using Crispr to edit a gene in the liver, and initial results show significant reductions in LDL cholesterol.
Gene editing could provide a lasting option for treating hereditary high cholesterol, replacing the need for long-term medication. [ more ]
Goodbye Statins? New Gene Editing Therapy Slashes High Cholesterol
A novel gene editing therapy using base editing lowered "bad" cholesterol levels in individuals with FH by as much as 55 percent.
The therapy could potentially provide a single-course treatment option for deep LDL lowering for decades without the need for daily pills or intermittent injections. [ more ]
U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders
The UK has authorized a gene-editing medicine for treating sickle cell disease and beta thalassemia, marking the first approval worldwide for a therapy based on CRISPR technology.
The therapy, known as Casgevy, involves editing a gene in a patient's stem cells to produce high levels of fetal hemoglobin.
Casgevy offers a potentially curative treatment option for these inherited blood disorders, reducing the need for chronic therapies and blood transfusions. [ more ]
Researchers developed a gene-editing technology that reduces 'bad' cholesterol
A gene-editing treatment called VERVE-101 showed promising results in reducing cholesterol levels in patients with hereditary conditions.
The treatment used CRISPR editing techniques to turn off a cholesterol-raising gene in the liver, resulting in lower levels of LDL-C.
While the therapy has the potential to disrupt current high cholesterol treatments, further studies and FDA approval are needed before it can be widely available. [ more ]